Activity of lumacaftor is not conserved in zebrafish Cftr bearing the major cystic fibrosis-causing mutation
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F508del-cystic fibrosis transmembrane conductance regulator (CFTR) is the major mutant responsible for cystic fibrosis (CF). ORKAMBI®, approved for patients bearing this mutant, contains lumacaftor (VX-809) that partially corrects F508del-CFTR's processing de... ...